Short G-rich oligonucleotides as a potential therapeutic for Huntington's Disease.

ABSTRACT:

Background

Huntington's Disease (HD) is an inherited autosomal dominant genetic disorder in which neuronal tissue degenerates. The pathogenesis of the disease appears to center on the development of protein aggregates that arise initially from the misfolding of the mutant HD protein. Mutant huntingtin (Htt) is produced by HD genes that contain an increased number of glutamine codons within the first exon and this expansion leads to the production of a protein that misfolds. Recent studies suggest that mutant Htt can nucleate protein aggregation and interfere with a multitude of normal cellular functions.

Results

As such, efforts to find a therapy for HD have focused on agents that disrupt or block the mutant Htt aggregation pathway. Here, we report that short guanosine monot

SUBMITTER: Skogen M 

PROVIDER: S-EPMC1609172 | biostudies-literature | 2006 Oct

REPOSITORIES: biostudies-literature